Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
REGN7999 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Part A High Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part A Low Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part A Placebo | PLACEBO_COMPARATOR | Randomized 2:2:1 |
| Part B High Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part B Low Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part B Placebo | PLACEBO_COMPARATOR | Randomized 2:2:1 |
| Name | Type | Description |
|---|---|---|
| REGN7999 | DRUG | Administered subcutaneous (SC) |
| Placebo | DRUG | Administered SC |
Key Inclusion Criteria: 1. Clinical diagnosis of NTDT as described in the protocol 2. IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2\* MRI at screening 3. Serum ferritin ≥ 300 ng/mL as described in the protocol Key Exclusion Criteria: 1. Hemoglobin ≤ 8 g/dL at screening 2. Any RBC transfusi...
REGN7999 is an investigational small molecule developed by Regeneron Pharmaceuticals for non-transfusion dependent beta-thalassemia, a blood disorder in which patients have iron overload without needing regular blood transfusions. It is designed to reduce iron accumulation and is being studied in a Phase 2 clinical trial.
REGN7999 targets TMPRSS6, a protein involved in regulating the body's iron balance. By acting on TMPRSS6, the drug is intended to lower iron levels in patients with non-transfusion dependent beta-thalassemia. The target is a protein, and REGN7999 is a small molecule.
Regeneron Pharmaceuticals, Inc. is developing REGN7999. Regeneron trades on the Nasdaq under the ticker REGN. The company is running the clinical program for the drug in non-transfusion dependent beta-thalassemia.
REGN7999 is in Phase 2 clinical development. It is investigational and has not been approved by the FDA. The ongoing Phase 2 study is evaluating the drug in adults with non-transfusion dependent beta-thalassemia.
REGN7999 is being studied in one active clinical trial, NCT06421636. This Phase 2 study is testing the safety, tolerability, and efficacy of REGN7999 injected under the skin in adults with non-transfusion dependent beta-thalassemia and iron overload. It uses MRI scans to measure iron levels and is currently active but not recruiting.
In the Phase 2 trial NCT06421636, REGN7999 is injected under the skin, or subcutaneously. The study is placebo-controlled, randomized, and double-blind, and it uses MRI scans to measure iron levels in the body. It enrolled 19 adult participants with non-transfusion dependent beta-thalassemia.